Protocol Summary

Protocol No.: OCR33522

Sponsor Protocol No.: NMTRC014

Protocol Title.: Neuroblastoma Maintenance Therapy Trial

Principal Investigator: Lagmay, Joanne

Objective: Difluoromethylornithine (DFMO) will be used in an open label, single agent, multicenter, study for patients with neuroblastoma in remission. In this study subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 750 mg/m2 ± 250 mg/m2 BID (strata 1, 2, 3, and 4) OR 2500 mg/m2 BID (stratum 1B) on each day of study. This study will focus on the use of DFMO in high risk neuroblastoma patients that are in remission as a strategy to prevent recurrence.

Phase: Phase II

Age Group: Both

Age: 1 Year - 30 Years

Gender: All

Scope: National

Treatment:

Experimental: Difluoromethylornithine (DFMO)
Subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 750 mg/m2 ± 250 mg/m2 BID (strata 1, 2, 3, and 4) OR 2500 mg/m2 BID (stratum 1B) on each day of study.

Detailed Eligibility:

Inclusion Criteria: - All patients must have a pathologically confirmed diagnosis of neuroblastoma, 50% and patients must have a life expectancy of ≥ 2 months. - All clinical and laboratory studies for organ functions to determine eligibility must be performed within 7 days prior to enrollment unless otherwise indicated below. - Patients must have adequate organ functions at the time of registration: - Hematological: Total absolute phagocyte count ≥1000/μL - Liver: Subjects must have adequate liver function - Renal: Adequate renal function - Females of childbearing potential must have a negative pregnancy test. Patients of childbearing potential must agree to use an effective birth control method. Female patients who are lactating must agree to stop breast-feeding. - Written informed consent in accordance with institutional and FDA (food and drug administration) guidelines must be obtained from all subjects (or patients' legal representative). Exclusion Criteria: - BSA (Body Surface Area) of <0.25 m2. - Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation. - Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are not eligible. Subjects must have fully recovered from hematological and bone marrow suppression effects of prior chemotherapy. - Infection: Subjects who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator. - Subjects who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.

Applicable Conditions:

  • Brain and Nervous System
  • Participation Institution:

  • UF Gainesville : Ashley Bayne
  • Contact:
    Ashley Bayne
    Phone: +1 352-294-8745
    Email: abayne@ufl.edu

    More Information: View study listing on ClinicialTrials.gov http://www.clinicaltrials.gov/ct2/show/NCT02679144